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Results for “Pyruvate Kinase Deficiency”

17 trials

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Showing 17 of 17 results

Large-scale testing (Phase 3)Active Not RecruitingNCT05175105
What this trial is testing

Evaluate the Efficacy and Safety of Mitapivat in Pediatric Participants With Pyruvate Kinase Deficiency (PKD) Who Are Not Regularly Transfused, Followed by a 5-Year Extension Period

Who this might be right for
Pediatric Pyruvate Kinase DeficiencyPediatric Hemolytic Anemia
Agios Pharmaceuticals, Inc. 30
Not applicableEnded earlyNCT04964323
What this trial is testing

Pyruvate Kinase (PK) Deficiency Global Longitudinal Registry: Patient-Reported Outcomes (PRO)

Who this might be right for
Pyruvate Kinase DeficiencyPyruvate Kinase Deficiency AnemiaAnemia+1 more
Agios Pharmaceuticals, Inc. 15
Post-approval studies (Phase 4)Enrolling By InvitationNCT05777993
What this trial is testing

Provide Continued Access to Mitapivat for Participants Who Previously Completed an Agios-Sponsored Mitapivat Study

Who this might be right for
Pyruvate Kinase DeficiencyAnemia, Hemolytic
Agios Pharmaceuticals, Inc. 6
Large-scale testing (Phase 3)Study completedNCT03559699
What this trial is testing

A Study Evaluating the Efficacy and Safety of AG-348 in Regularly Transfused Adult Participants With Pyruvate Kinase Deficiency (PKD)

Who this might be right for
Pyruvate Kinase DeficiencyAnemia, Hemolytic
Agios Pharmaceuticals, Inc. 27
Early research (Phase 1)Study completedNCT04105166
What this trial is testing

Gene Therapy for Pyruvate Kinase Deficiency (PKD)

Who this might be right for
Pyruvate Kinase Deficiency
Rocket Pharmaceuticals Inc. 4
Not applicableLooking for participantsNCT06213402
What this trial is testing

RADeep Multicenter European Epidemiological Platform for Patients Diagnosed With Rare Anemia Disorders (RADs)

Who this might be right for
Sickle Cell DiseaseThalassemiaHemolytic; Anemia, Hereditary, Due to Enzyme Disorder+6 more
Hospital Universitari Vall d'Hebron Research Institute 32,564
Large-scale testing (Phase 3)Study completedNCT03853798
What this trial is testing

Extension Study of AG-348 in Adult Participants With Pyruvate Kinase Deficiency Previously Enrolled in AG-348-006 or AG348-C-007

Who this might be right for
Pyruvate Kinase Deficiency
Agios Pharmaceuticals, Inc. 90
Early research (Phase 1)Not Yet RecruitingNCT07612345
What this trial is testing

High-Dose Vitamin C in G6PDA and Pyruvate Kinase Deficiency: A Safety Study

Who this might be right for
Pyruvate Kinase DeficiencyGlucose 6 Phosphate Dehydrogenase Deficiency
University of Utah 3
Not applicableActive Not RecruitingNCT03481738
What this trial is testing

Pyruvate Kinase Deficiency Global Longitudinal Registry

Who this might be right for
Pyruvate Kinase Deficiency
Agios Pharmaceuticals, Inc. 500
Testing effectiveness (Phase 2)Study completedNCT02476916
What this trial is testing

AG-348 in Adult Participants With Pyruvate Kinase (PK) Deficiency

Who this might be right for
Pyruvate Kinase Deficiency
Agios Pharmaceuticals, Inc. 52
Not applicableStudy completedNCT04902833
What this trial is testing

Acquired Pyruvate Kinase Deficiency In Clonal Myeloid Neoplasms

Who this might be right for
Pyruvate Kinase DeficiencyPyruvate Kinase Deficiency AnemiaHereditary Hemolytic Anemia+8 more
Massachusetts General Hospital 18
Not applicableStudy completedNCT04995315
What this trial is testing

Pyruvate Kinase Deficiency Global Longitudinal Registry Substudy of Protocol AG348-C-008

Who this might be right for
Pyruvate Kinase Deficiency
Agios Pharmaceuticals, Inc. 13
Large-scale testing (Phase 3)Active Not RecruitingNCT05144256
What this trial is testing

Evaluate the Efficacy and Safety of Mitapivat in Pediatric Participants With Pyruvate Kinase Deficiency (PKD) Who Are Regularly Transfused, Followed by a 5-Year Extension Period

Who this might be right for
Pediatric Pyruvate Kinase DeficiencyPediatric Hemolytic Anemia
Agios Pharmaceuticals, Inc. 49
Testing effectiveness (Phase 2)Temporarily pausedNCT06422351
What this trial is testing

Clinical Trial to Evaluate the Efficacy of Gene Therapy for Pyruvate Kinase Deficiency

Who this might be right for
Pyruvate Kinase Deficiency
Rocket Pharmaceuticals Inc. 10
Large-scale testing (Phase 3)Study completedNCT03548220
What this trial is testing

Evaluate Efficacy and Safety of AG-348 in Not Regularly Transfused Adult Participants With Pyruvate Kinase Deficiency (PKD)

Who this might be right for
Pyruvate Kinase DeficiencyAnemia, Hemolytic
Agios Pharmaceuticals, Inc. 80
Not applicableStudy completedNCT03866590
What this trial is testing

Pyruvate Kinase Deficiency Epidemiological Study (PIECE)

Who this might be right for
Pyruvate Kinase Deficiency
CENTOGENE GmbH Rostock 75
Not applicableStudy completedNCT02053480
What this trial is testing

Pyruvate Kinase Deficiency Natural History Study

Who this might be right for
Pyruvate Kinase DeficiencyCongenital Non-Spherocytic Hemolytic Anemia
Boston Children's Hospital 254